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Korean J Pediatr 2018 February;61(2) :53-58.
Published online 2018 February 15.       
Excellent treatment outcomes in children younger than 18 months with stage 4 MYCN nonamplified neuroblastoma
Chiwoo Kim1, Young Bae Choi2, Ji Won Lee1, Keon Hee Yoo1, Ki Woong Sung1, Hong Hoe Koo1
1Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
2Department of Pediatrics, Chung-Ang University Hospital, Seoul, Korea
Corresponding Author: Young Bae Choi ,Tel: +82-2-6299-1476, Fax: +82-2-6264-2167, Email:
Copyright © 2018 by The Korean Pediatric Society
Purpose: Although the prognosis is generally good in patients with intermediate-risk neuroblastoma, no consensus has been reached on the ideal treatment regimen. This study analyzed treatment outcomes and toxicities in patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma.
Methods: We retrospectively analyzed 20 patients younger than 18 months newly diagnosed with stage 4 MYCN nonamplified neuroblastoma between January 2009 and December 2015. Patients received 9 cycles of chemotherapy and surgery, with or without local radiotherapy, followed by 12 cycles of differentiation therapy with 13-cis-retinoic acid. Chemotherapy consisted of alternating cycles of cisplatin, etoposide, doxorubicin, and cyclophosphamide (CEDC) and ifosfamide, carboplatin, and etoposide (ICE) regimens.
Results: The most common primary tumor site was the abdomen (85%), and the most common metastatic sites were the lymph nodes (65%), followed by the bones (60%), liver (55%), skin (45%), and bone marrow (25%). At the end of induction therapy, 14 patients (70%) achieved complete response, with 1 achieving very good partial response, 4 achieving partial response, and 1 showing mixed response. Nine patients (45%) received local radiotherapy. At a median follow-up of 47 months (range, 17–91 months), none of these patients experienced relapse, progression, or secondary malignancy, or died. Three years after chemotherapy completion, none of the patients had experienced grade 3 late adverse effects.
Conclusion: Patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma showed excellent outcomes, without significant late adverse effects, when treated with alternating cycles of CEDC and ICE, followed by surgery and differentiation therapy.
Keywords: Neuroblastoma | Infant | Neoplasm metastasis | N-myc proto-oncogene protein
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